Netherton's Syndrome

Genetically modifying stem cells is being investigated as possible treatments for the genetic condition - Netherton's Syndrome. Click on the link provided to find out more about the trial. It remains too early to know if the approach will be suitable. We need evidence collected in clinical trials to determine if a proposed stem cell treatment is both safe and effective.

tagged as Netherton's Syndrome

  • Gene Therapy for Netherton Syndrome Location: UK
    Overview: This trial seeks to investigate the effect of skin grafts in five patients with Netherton's syndrome, a genetic condition where the patient’s skin becomes extremely dry and easily inflamed. In this study, the patient’s skin stem cells will be harvested and then genetically manipulated in the laboratory to correct the mutation (in the SPINK5 gene). These corrected cells will be cultured in the laboratory before being grafted to the patient, where it is hoped that the transplanted cells will produce the proteins lacking in the condition (LEKT1) and benefit the patient. The trial is being conducted at Great Ormond Street Children's Hospital in London. The trial is expected to finish in July 2016.
    Link to Clinical Registry
    Trial Design: Safety and Efficacy Study
    Status: Not yet started
    Stem Cell: Stem Cells (unspecified)